Abstract
Managed access agreements provide a crucial mechanism whereby real-world data can be collected systematically to reduce uncertainty around available clinical and economic data, whilst providing the opportunity to identify patient sub-populations who are most likely to benefit from a new treatment. This manuscript aims to share learnings from the first managed access agreement, which was initiated following positive conditional approval in 2015 from the National Institute for Health and Care Excellence (NICE) for elosulfase alfa, an enzyme replacement therapy for the treatment of mucopolysaccharidosis type IVA (MPS IVA). This managed access agreement enabled the collection of comprehensive real-world data for patients with MPS IVA, with results demonstrating that patients starting elosulfase alfa treatment showed gains similar to those seen in the pivotal trial for outcomes including endurance, respiratory and cardiac function, pain, quality of life measures and urinary keratan sulfate levels. In addition, former trial patients continued to see benefits in both clinical assessments and quality of life/activities of daily living nine years after beginning treatment. Key strengths of the process included recruitment of a high proportion of MPS IVA patients treated in England (72/89 known eligible patients) with a wide range of ages (2–58 years). Participation of a patient organisation (the MPS society) ensured that the patient voice was present throughout the process, whilst a contract research organisation (Rare Disease Research Partners) ensured that patients were represented when interpreting agreement criteria and during patient assessment meetings. Longer-term follow-up will be required for several MPS IVA outcomes (e.g. skeletal measures) to further reduce uncertainty, and continued follow-up of patients who had stopped treatment was found to be challenging. The burden associated with this managed access agreement was found to be high for patients, physicians, patient organisations, NHS England and the manufacturer, therefore costs and benefits of future agreements should be considered carefully before initiation. Through evaluation of the strengths and limitations of this process, it is hoped that learnings from this managed access agreement can be used to inform future agreements.
Highlights
IntroductionInsufficient evidence and high unit costs often prevent orphan drugs from meeting health technology assessment (HTA) cost-effective requirements, leading to restricted access to potentially lifechanging treatments for patients with rare diseases [5]
Specialised technology (HST) evaluations are conducted by the National Institute for Health and Care Excellence (NICE) to make recommendationsStevens et al Orphanet J Rare Dis (2021) 16:394 of natural history, resource use and quality of life data [4]
Products which receive a conditional approval in the Highly specialised technology (HST) programme may be required to generate further evidence through a managed access agreement (MAA) in order for reimbursement to be considered in the long term [7]
Summary
Insufficient evidence and high unit costs often prevent orphan drugs from meeting health technology assessment (HTA) cost-effective requirements, leading to restricted access to potentially lifechanging treatments for patients with rare diseases [5]. To address these challenges, the HST programme uses a wider evaluation framework than STAs or MTAs, taking into consideration the nature of the condition in question and the technology’s perceived value for money. Products which receive a conditional approval in the HST programme may be required to generate further evidence through a managed access agreement (MAA) in order for reimbursement to be considered in the long term [7]
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